Development of Orphan Drugs through Repurposing
Ramaiah Muthyala, Associate Professor, University of Minnesota
From start to finish, on average a new experimental drug for common diseases takes 10-15 years and costs $1-1.5 billion. While more than 5000 compounds enter pre-clinical trials only one compound makes to the market. Orphan drug discovery is not different from discovering medicines for common diseases. The market potential for orphan drugs is not large due to smaller patient populations. Due to such risks pharmaceutical companies are shying away from developing drugs for rare diseases. To encourage the drug companies to developing orphan products, the US Orphan Drug Act (ODA) provides several incentives, for example, financial assistance of various types, market exclusivity, among others. Repositioning has significant commercial value as it extends the markets for a repurposed chemical at lower financial risk. The benefits of repositioning includes the easy availability of small molecules, toxicology studies that contribute significantly to time saving and cost-effective research etc. Combining the benefits of ODA with finding new uses for existing drugs has motivated several drug companies developing orphan products. In the past five years, there have been several journal review articles and at least a dozen conferences highlighting the potential value of repurposing for orphan drugs. Development of biopharmaceuticals is entering a new era due to new discoveries from human genome which offers new treatments for many rare diseases. According to US FDA, in the last five years, one third of all new drugs were for rare diseases in spite of challenges with clinical trials for rare diseases. Due to near empty new drug pipeline, expired patent portfolios, consumer pressure for lower drug prices, major pharma companies are consolidating their resources by mergers, cross-licensing, leaving common disease space to rare diseases space. In this presentation, new strategies highlighting the novel technologies, lead generation tactics, product development.
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Add to Calendar ▼2014-06-17 00:00:002014-06-18 00:00:00Europe/LondonTrack 1SELECTBIOenquiries@selectbiosciences.com
